The platform focuses on developing innovative therapies for β-thalassemia and other anemias based on the autologous hematopoietic stem cells through gene editing.

The platform develops the allogeneic CAR-T therapies for cancer, that is to remove receptors and proteins that can cause adverse reactions such as GvH and HvG on T cells of a healthy donor.

The platform develops therapeutics for Hurler syndrome, the most severe subtype of mucopolysaccharidosis type I, diseases related to liver, muscles and nerve system, etc. based on the RNA base editing technology LEAPER™.

Based on high-throughput gene editing technology, we identify the novel functional biomarkers or novel combination of targeted therapy via synthetic lethal screen and develop the innovative targeted therapies for solid tumor.